RNA-based therapies, and siRNAs in particular, have attractive therapeutic potential for
cancer treatment due to their ability to silence genes that are imperative for tumor …

Cardiovascular disease (CVD) has overtaken infectious illnesses as the leading cause of
mortality and disability worldwide. The pathology that underpins CVD is atherosclerosis …

Cancer is the second most frequent cause of death worldwide, with 28.4 million new cases
expected for 2040. Despite de advances in the treatment, it remains a challenge because of …

Efficient delivery of genetic material into cells is a critical process to translate gene therapy
into clinical practice. In this sense, the increased knowledge acquired during past years in …

Synthetic biology holds great promise to improve the safety and efficacy of future gene and
engineered cell therapies by providing new means of endogenous or exogenous control of …

Accelerating the scale up of adeno-associated virus (AAV) manufacture is highly desirable
to meet the increased demand for gene therapies. However, the development of …

Abstract Mucopolysaccharidosis IV A (MPS IVA) is a lysosomal disorder caused by
mutations in the GALNS gene. Consequently, the glycosaminoglycans (GAGs) keratan …

Since its discovery as a genome editing tool, the clustered regularly interspaced short
palindromic repeats and CRISPR-associated protein 9 (CRISPR/Cas9) system has opened …

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Lysosomal storage disorders (LSD) are a group of rare life-threatening diseases caused by
a lysosomal dysfunction, usually due to the lack of a single enzyme required for the …